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A Phase I Study of the CDK4/6 Inhibitor Ribociclib (LEE011) in Pediatric Patients with Malignant Rhabdoid Tumors, Neuroblastoma, and Other Solid TumorsIn this. i study the MTD and RP2D, safety, PK, and preliminary activity of single-agent ribociclib were investigated in patients with neuroblastoma.
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Contemporary survival endpoints: An international diffuse intrinsic pontine glioma registry studyThis study defines PFS and OS, and is the first describe post-progression survival in a large cohort of children with DIPG.
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Infective respiratory syncytial virus is present in human cord blood samples and most prevalent during winter monthsHuman respiratory syncytial virus (RSV) remains the most common cause of severe lower respiratory tract disease amongst infants, and continues to cause annual epidemics of respiratory disease every winter worldwide.
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The collective impact of rare diseases in Western Australia: An estimate using a population-based cohortThis cohort study provides new evidence of a disparity between the proportion of the population with rare diseases and their combined health-system costs
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Prevalence of microcephaly in an Australian population-based birth defects register, 1980-2015We identified a high proportion of cases without known cause, highlighting the need for clinicians to carefully investigate all possibilities, including emerging infections.
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In vitro drug susceptibility of two strains of the wildlife trypanosome, Trypanosoma copemani: A comparison with Trypanosoma cruziCompared the in vitro susceptibility of two strains of Trypanosoma copemani and one strain of T. cruzi against drugs that show trypanocidal activity
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Investigating self-efficacy, disease knowledge and adherence to treatment in adolescents with cystic fibrosisadolescents with cystic fibrosis are often sub-optimal with adherence to prescribed treatement. We aim to investigate multiple factors with patient and parents
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Mental health problems among 4–17-year-olds with hearing problems: results from a nationally representative studyClinicians caring for children and young people with hearing problems should be alert for heightened risk of specific mental health problems based on age and the nature of hearing problems.
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The effect of endotracheal tubes versus laryngeal mask airways on perioperative respiratory adverse events in infants: A randomised controlled trialEvaluating the difference of the effects of Laryngeal mask airways compared to endotracheal tubes in older children (>1 year) in incidents of PRAE in infants.
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Early lung surveillance of cystic fibrosis: what have we learnt?Newborn screening (NBS) for cystic fibrosis (CF) provides an opportunity to commence management and therapeutic interventions significantly earlier